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ErdoğanGenome editing technologies have demonstrated significant potential for treating diseases caused by various molecular changes.These include cancer, cystic fibrosis, diabetes, sickle cell anemia, heart diseases, and rare genetic disorders. 1,2Genome editing technologies also offer potential solutions for neurodegenerative diseases such as Alzheimer's and Huntington's disease and neuromuscular disorders such as Duchenne's muscular dystrophy, spinal muscular atrophy, and amyotrophic lateral sclerosis.The leading genome editing technologies are zinc-finger nucleases, transcription activatorlike effector nucleases, and clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9).Among these technologies, CRISPR/Cas9 is the most widely used due to its versatility, effectiveness, efficiency, and ease of application. 3his groundbreaking genome manipulation tool is revolutionizing scientific research, medical diagnostics, and therapeutic applications.Major advancements in editing efficacy have propelled genome editing strategies to phase 3 human clinical trials.However, the CRISPR technology is associated with some challenges such as high costs, ineffective delivery methods for specific targets, and poor efficiency of the genome editing processes, particularly due to offtarget effects. 3,
Suat Erdoğan (Fri,) studied this question.
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